Ipsen's Resubmission to the US Food & Drug Administration is Accepted

Palovarotene will receive an expedited review with a PDUFA date of December 29, 2022

We’re pleased to share breaking news from IpsenTheir resubmission to the US Food and Drug Administration (FDA) has been accepted for priority review. 

It is important to remember that this does NOT mean that palovarotene has been approved as a prescribed medication for FOP; it means that it is being reviewed and considered by regulators in the United States.

Regulatory submission is the process through which pharmaceutical companies submit the information about their proposed drug treatment to a regulatory agency for review and a decision on approval. Acceptance of Ipsen’s regulatory submission package means that the FDA has taken a preliminary look at the data and feels that it is sufficient to conduct a thorough review and make a decision as to whether palovarotene can be prescribed for the treatment of FOP

Ipsen has been granted priority review by the FDA. FDA priority review designation means their goal is to take action on an application within 6 months (compared to 10 months under standard review). 

The FDA’s PDUFA date is December 29, 2022. PDUFA is the date by which the FDA is expected to deliver its decision on whether or not to approve Ipsen’s New Drug Application (NDA) for palovarotene as a treatment for FOP.

It is important to remember that each country has its own version of the FDA and drugs must be approved in each country in order for physicians located there to prescribe them.

Decisions on whether or not insurance companies or national health services will pay for a drug will only be made following approval. This decision process is ongoing in Canada where Health Canada gave regulatory approval to SohonosTM (palovarotene capsules) in January 2022. Learn more.

Any decision as to which patients (e.g. by age group) can be prescribed a drug will be made during the review process.

Ipsen's press release notes that their new drug application package for palovarotene was submitted to the European Medicines Agency (EMA) in 2021 and in June 2022 they responded to questions raised by the EMA. The press release goes on to say that they anticipate additional applications to other regulatory agencies in due course.

We are grateful to each of you that have participated in the Ipsen (formerly Clementia) Natural History Study and the MOVE Trial. Without your support and involvement, this critical research would not have advanced. The same is true for those participating in the FOP Registry and other studies and trials, you are serving in a role that only you can fill. Thanks to each of you for advancing FOP research!

Read Ipsen’s full press release.

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